Late effects in patients with Fanconi anemia following allogeneic hematopoietic stem cell transplantation from alternative donors Journal Article

Authors: Praveen, A.; Friedman, D. N.; Sklar, C.; Oeffinger, K.; Castiel, M.; Kearney, J.; Singh, B.; Prockop, S. E.; Kernan, N. A.; Scaradavou, A.; Kobos, R.; Curran, K.; Ruggiero, J.; Zakak, N.; O'Reilly, R. J.; Boulad, F.
Article Title: Late effects in patients with Fanconi anemia following allogeneic hematopoietic stem cell transplantation from alternative donors
Abstract: Hematopoietic stem cell transplantation (HSCT) is curative for hematological manifestations of Fanconi anemia (FA). We performed a retrospective analysis of 22 patients with FA and aplastic anemia, myelodysplastic syndrome or acute myelogenous leukemia who underwent a HSCT at Memorial Sloan Kettering Cancer Center and survived at least 1 year post HSCT. Patients underwent either a TBI- (N = 18) or busulfan- (N = 4) based cytoreduction followed by T-cell-depleted transplants from alternative donors. Twenty patients were alive at time of the study with a 5- and 10-year overall survival of 100 and 84% and no evidence of chronic GvHD. Among the 18 patients receiving a TBI- based regimen, 11 (61%) had persistent hemochromatosis, 4 (22%) developed hypothyroidism, 7 (39%) had insulin resistance and 5 (27%) developed hypertriglyceridemia after transplant. Eleven of 16 evaluable patients (68%), receiving TBI, developed gonadal dysfunction. Two patients who received a TBI- based regimen died of squamous cell carcinoma. One patient developed hemochromatosis, hypothyroidism and gonadal dysfunction after busulfan- based cytoreduction. TBI appears to be a risk factor for malignant and endocrine late effects in the FA host. Multidisciplinary follow-up of patients with FA (including cancer screening) is essential for early detection and management of late complications, and improving long-term outcomes.
Keywords: long-term survivors; adults; versus-host-disease; bone-marrow-transplantation; malignancies; regimen; total-body irradiation; cytoreductive; grafts; aplastic-anemia; chronic gvhd; severe
Journal Title: Bone Marrow Transplantation
Volume: 51
Issue: 7
ISSN: 0268-3369
Publisher: Nature Publishing Group  
Date Published: 2016-07-01
Start Page: 938
End Page: 944
Language: English
ACCESSION: WOS:000379540200010
DOI: 10.1038/bmt.2016.32
PMCID: PMC4968886
PUBMED: 26999465
Notes: Article -- Source: Wos
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MSK Authors
  1. Nancy Kernan
    384 Kernan
  2. Farid Boulad
    255 Boulad
  3. Charles A Sklar
    281 Sklar
  4. Susan E Prockop
    161 Prockop
  5. Bhuvanesh Singh
    209 Singh
  6. Mercedes Castiel
    18 Castiel
  7. Kevin Oeffinger
    263 Oeffinger
  8. Kevin Joseph Curran
    59 Curran
  9. Nicole Zakak
    11 Zakak
  10. Richard O'Reilly
    487 O'Reilly
  11. Rachel Kobos
    65 Kobos
  12. Julia A Kearney
    26 Kearney